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Examining blood antibody levels and biomarkers in patients with late-onset Pompe disease

A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease

Not applicable Interventional Astellas Pharma Inc · NCT06150820

This study is trying to see how blood antibodies and other markers change in people with late-onset Pompe disease over two years to understand their immune response to existing treatments.

Quick facts

PhaseNot applicable
Study typeInterventional
Enrollment100 (estimated)
Ages16 Years to 69 Years
SexAll
SponsorAstellas Pharma Inc Industry-sponsored
Locations51 sites (Irvine, California and 50 other locations)
Trial IDNCT06150820 on ClinicalTrials.gov

What this trial studies

This study investigates the levels of antibodies and biomarkers in the blood of individuals diagnosed with late-onset Pompe disease (LOPD). Participants will provide blood and urine samples over approximately two years to better understand the immune response to enzyme replacement therapy. The study aims to gather data without administering any investigational drugs, focusing instead on the natural progression of the disease and the body's response to existing treatments. Participants can withdraw from the study at any time.

Who should consider this trial

Good fit: Ideal candidates include individuals with a documented clinical diagnosis of late-onset Pompe disease who are either naïve to enzyme replacement therapy or have been on it for at least six months.

Not a fit: Patients who have previously received an AAV-related product or are currently involved in other interventional studies may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of immune responses in Pompe disease, potentially leading to improved treatment strategies.

How similar studies have performed: While this approach is focused on observational data collection, similar studies have shown promise in understanding immune responses in genetic conditions.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant has a documented clinical diagnosis of LOPD.
* Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]).
* Participant is willing and able to comply with study visits and procedures.
* Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study.

Exclusion Criteria:

* Participant previously received an AAV-related product (any serotype).
* Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
* Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility).
* Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility).
* Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.

Where this trial is running

Irvine, California and 50 other locations

+1 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Pompe DiseaseAntibodies to AAV8Biomarkers
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.